A recent FDA advisory panel recommended the approval of 2 new agents in a novel class of cholesterol lowering drugs known as PCSK-9 inhibitors. What makes this remarkable is that these drugs illustrate all the promise and pitfalls of modern pharmaceutical development.

First, a little science. The target of the new drugs – a protein named proprotein convertase subtilisin/kexin type 9 (PCSK-9) – was discovered in 2001. Two years later, investigators reported that “gain-of-function” mutations in the gene that codes for PCSK-9 were associated with familial hypercholesterolemia and high rates of atherosclerotic vascular disease. Mutations of the gene that led to reductions in the function of PCSK-9 were associated with low LDL-cholesterol levels, and a lower incidence of vascular disease. That made the compelling case that PCSK-9 had a counter-regulatory function in LDL-cholesterol metabolism, so that interfering with its function would lead to lower cholesterol levels.

Author

This blog isn’t about sharing information; it’s about starting conversations. And since good conversations require good listening, I decided to call this blog “Auscultation.” Ira Nash, MD, FACC, FAHA, FACP

The views expressed here are solely the personal views of Ira Nash, MD and do not necessarily represent the policy or position of Northwell Health Physician Partners, Northwell Health or any of their affiliates, employees or physicians.

About Ira Nash, MD

Ira Nash, MD is the Executive Director of Northwell Health Physician Partners, and Senior Vice President of Northwell Health, and a professor of Cardiology and Population Health at Hofstra Northwell School of Medicine.